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Article

Expression of a Truncated Cystic Fibrosis Transmembrane Conductance Regulator with an AAV5-pseudotyped Vector in Primates

13 Feb 2007

Abstract excerpt

Gene therapy using recombinant adeno-associated virus (rAAV2) vectors for cystic fibrosis has shown gene transfer and remarkable safety, yet indeterminate expression. A new construct has been characterized with a powerful exogenous promoter, the cytomegalovirus enhancer/chicken beta-actin promoter, driving a truncated CF transmembrane conductance regulator (CFTR), pseudotyped in an AAV5 viral coat. Our goal is to...

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PMID: 17299412
DOI: 10.1038/sj.mt.6300059

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